Information for patients considering registering with a patient registry
Before you agree to register in a patient registry it is important that you understand what is involved and what will be done with the information you provide. This page contains answers to some of the questions you might have. The registry coordinators in your country will also be able to answer your questions about your own national registry.
For conditions that are ultra-rare then there may only be an international registry available, where there is only one registry for that condition regardless of where you live.
What is a patient registry?
Simply put, a patient registry is a database or collection of information about people affected by a particular condition. For the neuromuscular registries they are used to collect genetic and clinical information about people affected by neuromuscular diseases (NMDs).
Why is a patient registry needed?
Scientific advances over recent years have led to substantial changes in the treatment of many neuromuscular diseases. New therapeutic strategies are being developed and, for some of these treatments, trials are already underway. Several new therapeutic strategies for neuromuscular diseases target specific genetic defects, some of which are so rare that only a few patients in the world will have the right profile for a clinical trial. When a clinical trial is being planned, it is very important that patients suitable for that trial can be found and contacted quickly.
The best way of ensuring this can happen is to make sure that patients’ details are all collected together in a single database or ‘registry’ that contains all the information that researchers will need, including each patient’s particular genetic defect and other key information about their disease.
Which patients are eligible?
All patients with a confirmed diagnosis (or pending diagnosis) in one of the conditions for which registries exist are eligible for inclusion. For upload into the TREAT-NMD Global Registry, diagnosis must be confirmed via genetic testing results.
Which diseases has TREAT-NMD set up Global Registries for?
TREAT-NMD Global Registries have been set up for spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD). Global Registries for other conditions are in preparation.
How can I join a TREAT-NMD Global Registry specific to my condition and where will my data go?
First, by using the link at the end of this page, check if there is a registry for your condition in your country. If you agree to register in your own national registry, you should read the patient information and sign the consent form associated with your own national registry.
The registry questionnaire asks you for some personal data and some information about your disease. In some countries, you can enter this information yourself online or on a paper form, while in others, you and your doctor will discuss it together at a consultation. The information you provide will be entered into a national registry in your own country. Your data will be stored securely and no unauthorized people will be able to gain access to any information about you. Your data will be assigned a unique code, and the data about all patients in each country’s national registry will then be fed into the TREAT-NMD Global Registry, without including your personal information. When planning clinical trials, researchers can ask the TREAT-NMD Global Registry to look at the data to find participants eligible for their trial, based on the patients’ clinical and genetic data. Only researchers who have been approved by their own local ethics committee and by the TREAT-NMD Global Database Oversight Committee are allowed to do this.
In the TREAT-NMD Global Registry, your data will only be identified by an anonymous code, not by your name. This means that people accessing the registry at the global level will not be able to find out your personal information (name, address etc.), but only the information they need about your disease that will help them decide whether you might be suitable for the trial. If they think you meet the criteria and might benefit from the trial, they will contact the person in charge of your national registry. Staff working for the national registry will ‘de-code’ the data to find out your personal details and will contact you to give you information about the trial or about any other issues relevant to your disease. They will not give your name or any personal information to the researchers. If you are interested in the information you receive about a particular clinical trial, you will be given information about how you can contact the researchers running the trial. If you decide to take part in the trial, you will need to review and sign a separate consent form. You are completely free to make your own decision about any trial we inform you about. If you decide not to take part in a particular trial, your data will still be kept in the registry and we will continue to inform you about other trials unless you tell us not to. Please note that if we tell you about the existence of a trial, this does not imply that we endorse it.
How can I update my data if it changes?
To make sure that the data in the registry is correct and up to date, it is essential that it is updated regularly. To do this, your national registry will contact you at least once a year asking you to tell it about any changes in your medical condition. It is also important to inform the registry about any major changes in your details that might occur in the period between updates, for example change of address or loss of ability to walk.
Who will have access to my medical records?
Staff in charge of your national registry will have access to the information you provide. They might also need to gain access to your medical records to obtain information necessary to the project (for example, they might need to ask your geneticist to give them a copy of your genetic report). If this happens, you will be asked by your own national registry to consent to this.
How will I be identified in the registry?
Your personal details (name, address etc.) have to be stored in your national registry so that they can contact you if they need to inform you about possible clinical trials or anything else that might be relevant to your disease. This data will be stored in a secure manner and your records will be assigned a unique code. When the national registry transfers your data to the TREAT-NMD Global Registry, it will not transfer any of your personal details, and your records will only be identifiable by the code they have been assigned. Researchers accessing the TREAT-NMD Global Registry therefore cannot identify you personally from the information they have access to. Only the person in charge of your own national registry or a person explicitly appointed by them will be able to ‘de-code’ the data to get access to your personal details.
Where will my data be kept and will it be kept confidential?
Your data will be kept for an indefinite period under the responsibility of the coordinator of your national registry. Creating a registry requires the existence of a file containing a patient’s personal and medical data. This file will be subject to the regulations on data protection (GDPR/UK data protection act, and any other local laws depending on the country). All information kept in the national registry will be treated confidentially. If any research or other documents based on data from the registries is published, this research will never identify you by name.
Third parties wishing to have access to data in the TREAT-NMD Global Registry (such as researchers or companies planning clinical trials or conducting research on new therapies) will only be given anonymous information identifiable only by a code in the form of a report. They will never be given direct access to the registry database. Before they are granted access even to this anonymous information, they have to have the approval of an Ethics Committee. Your data will not be made available to employers, governmental organizations, insurance companies or educational institutions, nor to your spouse, other members of your family or your doctor.
Will you ever give my name and address to a company running a clinical trial?
No, a national registry will never pass on any personally identifiable data to anyone outside the registry. If a company asks to find patients who might be suitable for their trial, you will be sent a letter giving you details about the trial and the name of the person to contact if you are interested (usually the doctor who is running the trial at the centre nearest you). That is the only legal way that patients can be recruited for trials, and we think it is also the best way, because you have time to consider whether it is something you are interested in without being put under any pressure.
How will I benefit from registering?
Registries are intended as a public service for the benefit of patients living with neuromuscular diseases. You will not receive any payment or any other financial benefit as a result of submitting your data to the registry. The results of research facilitated by the registry may be patentable or may have commercial potential. However, you will not receive patent rights and will not receive financial benefits from future commercial development. Nevertheless, there may be other benefits to participating, including the following:
You will be informed if (on the basis of the information you provide) you might be a suitable candidate for a certain clinical trial.You will also be informed of any new information relating to your disease which might be of interest to you – for example if better ways of caring for patients with your condition have been identified.The data collected might also provide benefits to other patients with your disease, for example by revealing statistics on how many people across the world have the same condition, or providing information for researchers interested in the best standards of care for your disease.
I want to be involved in a clinical trial. If I register, is this guaranteed?
Although one of the main aims registries, particularly those affiliated with TREAT-NMD, is to make it easier for patients to be recruited for clinical trials, there is no guarantee that registering your details will ensure you will be involved in a clinical trial.
TREAT-NMD has no control over where companies decide to run their trials or which patients are enrolled – we can provide companies with information and advice, but it remains their own decision. However, it is important that you understand that even if a trial is happening in your country and the people in charge of the trial believe that you might be eligible for that trial, based on the data about you stored in the TREAT-NMD Global Registry, it is still possible that once they have assessed you fully, it will turn out that you do not meet the trial inclusion criteria after all.
I don't want to be involved in a clinical trial. Should I still register?
We hope you will be interested in registering even if you don’t want to take part in a trial. Your information will still be useful to researchers who are trying to find out more about people living with your disease, and you should still be provided you with other information that might be relevant to your disease.
Do I have to participate in a registry and can I withdraw if I change my mind?
Your participation in a registry is completely voluntary. National data protection laws grant you the right to access your own data and to rectify it or withdraw it completely at any time. Should you wish to withdraw your data from a registry you will be free to do so without having to provide any explanation. If you wish to withdraw, you should get in touch with the staff in charge of your own national registry.
Who should I contact if I have any questions?
If you would like any additional information about your own national registry, please contact the people in charge at the national registry. Information can be found ….
For information about the TREAT-NMD Global Registries, contact Professor Jan Verschuuren.